MHRA Grants ADvantage Therapeutics Innovative Licensing and Access Pathway (ILAP) Designation for Novel Lead Product AD04™ for Phase 2b Trial in Alzheimer’s DiseaseILAP Designation accelerates patient access and commercialization
MIAMI, April 05, 2023 (GLOBE NEWSWIRE) -- ADvantage Therapeutics, Inc. (“ADvantage” or “the Company”), which is developing therapies to treat neurodegenerative conditions with a central focus on Alzheimer’s Disease (AD), today announced that the Medicines and Healthcare products Regulatory Agency (MHRA), the United Kingdom National Competent Authority, has granted the Company’s lead compound AD04™ an Innovation Passport for the treatment of AD under the Innovative Licensing and Access Pathway (ILAP).
ILAP was established in 2021 to reduce the time to market for innovative medicines in the United Kingdom. According to the Agency, the ILAP combines the MHRA’s globally recognized strengths of independence and high standards of quality, safety, and efficacy, with improved efficiency and flexibility, readying the MHRA for a new era in medicines approvals in the UK. The designation allows for significant input and collaboration with the National Institute for Health and Care Excellence (NICE), the Scottish Medicines Consortium (SMC) and NHS England and NHS Improvement (NHSE&I). In addition, patients are also considered key partners and the patient voice is integrated at every development stage. The MHRA indicates that ILAP incorporates broad and inclusive concepts of innovation and patient need.
“We are delighted that MHRA has recognized the potential of AD04™ at same time we are launching our confirmatory trial,” said Jeffrey Madden, chief executive officer of ADvantage. “AD04™ may completely change the approach to the treatment of Alzheimer’s Disease. The ILAP designation creates an accelerated pathway to approval, commercialization, and patient access, by providing a more open dialogue with all the U.K. agencies, including NICE, which oversees access and integration of novel therapies into the National Health Service (NHS) and NHS Wales. We believe our novel immunotherapy for Alzheimer’s has a low manufacturing cost, simple storage, subcutaneous administration, and believe that forthcoming clinical studies will confirm safety, efficacy and tolerability. If proven and approved, the rapid introduction of AD04™ into the U.K. health system may support accelerated clinical adoption in other worldwide healthcare systems.”
The Phase 2b trial for AD04™ in the ILAP application will be a randomized, placebo-controlled, double-blind study to confirm proof of concept and establish the safety and efficacy of AD04™ in mild AD patients. The 12-month study will measure hippocampal volume as an objective/biomarker endpoint. Other measured outcomes include the Alzheimer’s Disease Assessment scale – cognition 13-item scale (ADAScog13), the Alzheimer’s Disease Cooperative Study – Activities of Daily Living Standards (ADCS-ADL), the Clinical Dementia Rating – sum of boxes (CDR-sb), the patient Quality of Life-Alzheimer’s Disease and the Neuropsychiatric Inventory (NPI).
A 2mg dose of AD04™ served as a control in a prior study of another compound, where it demonstrated a statistically significantly slower decline in outcomes (cognition, function and quality of life) than other arms of that trial. In addition, the AD04™ control group showed slower decline in MRI-measured hippocampal volume as a biomarker of AD progression. To date, preclinical studies have shown that AD04™ decreased the number of inflammatory microglial cells in the hippocampus of mouse models. Inflammation and hyperproliferation of microglial cells are pathological immunological events in the brain of Alzheimer’s patients.
Dr. Rudolph Tanzi, who serves as Chair of the Scientific Advisory Board at ADvantage Therapeutics, Inc. stated, “The democratization of Alzheimer’s disease treatments that are safe, effective, and affordable for all are of utmost importance. We hope this AD04 trial with ILAP designation will bring us one step closer,” he concluded.
The Innovative Licensing and Access Pathway (ILAP) aims to provide significant benefits to patients, the National Health Service (NHS) and the life sciences industry by delivering a safe and efficient route for accelerated access to new medicines, as well as encouraging growth of pharmaceutical research and investment in the UK. In addition to (MHRA, NICE, SMC, AWTTC), other partners within the pathway include NHS England and NHS Improvement (NHSEI) and the National Institute for Health Research (NIHR). The ILAP has for the first time facilitated collaboration between NICE, the AWTTC, and the SMC under a unified umbrella to support activities, provide scientific advice, and engage a variety of stakeholders in the Health Technology Assessment (HTA) evaluation of new technologies.
Product developers within the ILAP are given the ability to jointly consult all partners as required at each stage and discuss potential hurdles in advance for obtaining concurrent advice on navigating the path for successful market access. It requires applicants to meet three key criteria. The first is that the condition the medicinal product aims to treat is life-threatening or seriously debilitating or there is a significant patient or public health need.
Second, the medicinal product must fulfill one or more specific criteria, which include being an innovative medicine such as an advanced therapy medicinal product (ATMP) or new chemical or biological entity or novel drug device combination, a medicine being developed in a clinically significant new indication for an approved medicine, a medicine for rare disease and/or other special populations such as neonates and children, elderly, and pregnant women, or is a development aligning with the objectives for UK public health priorities such as the Chief Medical Officer, Department of Health and Social Care (DHSC), or Life Sciences Sector Deal (including those in Devolved Administrations, where appropriate).
Third, the medicinal product must have the potential to offer benefits to patients.
ADvantage Therapeutics is developing AD04™ as a novel immunotherapy for mild Alzheimer’s Disease. The compound has been used as an adjuvant in human and animal vaccination programs. In a previous trial, AD04™ serving as a control against another compound appeared to demonstrate statistically significantly slower decline over other treatment groups in cognitive and quality life clinical measures. AD04™ also showed slower decline in hippocampal volume as a biomarker.
The Company believes that rather than being limited to a specific aspect of AD pathology, such as amyloid beta or tau, AD04™ may address immunological mechanisms in the brain and at the periphery. The Company believes AD04™ may act as an immunomodulator, stimulating and/or regulating the immune system to reduce AD pathology.
About Alzheimer’s Disease
About 44 million people worldwide suffer from Alzheimer’s Disease, and it is the sixth leading cause of death in industrialized countries. In 2019, the World Health Organization estimated the total worldwide cost of dementia at $1.3 trillion and expects this cost to rise to $2 trillion by 2030. While the FDA provisionally approved an anti-amyloid-beta antibody, Aducanumab, in 2021, widespread coverage has not been granted, nor have European countries approved the product. The socio-economic burden of Alzheimer’s Disease is enormous. AD devastates the lives of patients and their families. They lose their memories and independence, and the loss of a loved relation leaves behind guilt, grief, and anger. Alzheimer’s Disease is a high unmet medical need as there are currently no disease-modifying drugs approved worldwide. The availability of a safe, effective, affordable drug would transform the life of an AD patient from accepting a debilitating disease to the retention of personality, independence, and dignity.
About ADvantage Therapeutics
Headquartered in the Wynwood neighborhood in Miami, ADvantage Therapeutics is developing therapies to treat neurodegenerative conditions with a central focus on Alzheimer’s disease. The Company’s lead compound AD04™ is an injectable therapy in the process of entering into a confirmatory Phase 2b clinical trials in Europe to evaluate safety and efficacy of the product in early Alzheimer’s Disease. The Company believes that AD04™ may act as an immunomodulator, stimulating and regulating the immune system to reduce AD pathology, rather than limiting therapy to the aggregation of the proteins present once pathology is present. The Company is also exploring additional approaches to mitigating neurodegenerative disease, which it believes will eventually have an overall impact on longevity.
About ADvantage Therapeutics GmbH
ADvantage Therapeutics GmbH, founded in 2021, is the Vienna BioCenter-based subsidiary of ADvantage Therapeutics, Inc., where the Company conducts early research and drug development. ADvantage is developing therapies to treat neurodegenerative conditions like Alzheimer’s Disease (AD) and is currently preparing for clinical trials in Europe and in the U.S.
Safe Harbor-Forward-Looking Statements
This press release may contain forward-looking statements, including statements of potential mode of action, potential clinical effect, potential safety, and ADvantage’s potential clinical development program and pipeline program. ADvantage is in the early stages of developing and testing its AD04™ compound and may not receive regulatory approval to conduct the contemplated Phase 2b trial. The described clinical effect of our lead compound AD04™ is primarily based on results of a Phase 2 study designed to test a different compound. The described results need to be reproduced for proof of concept, might not be representative of larger scale clinical trials, and do not guarantee future regulatory approval or clinical success. Any preclinical results presented here are interim. The mechanism of action of AD04™ as potentially determined in our future investigations, particularly in future clinical trials with patients with Alzheimer’s Disease, might differ from the one presented.
For ADvantage Therapeutics
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Jeffrey Madden CEO
David Buchsbaum CFO
JQA Partners, Inc.